FDA OKs Sanofi’s Rare-Disease Drug Cablivi
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The US Food and Drug Administration (FDA) has approved Sanofi’s Cablivi (caplacizumab-yhdp) for treating thrombotic thrombocytopenic purpura (aTTP), a rare disease that causes blood clotting. The drug was approved in combination with plasma exchange and immunosuppression for treating aTTP.

Sanofi gained Cablivi through its $4.8-billion acquisition of Ablynx, a Ghent, Belgium-based biopharmaceutical company focused on developing and commercializing proprietary therapeutic proteins based on single-domain antibody fragments. Sanofi acquired Ablynx in June 2018. At that time of the acquisition, Sanofi said the acquisition of Ablynx continues its focus on technologies addressing multiple disease targets with single multi-specific molecules. Cablivi is part of Sanofi’s rare blood disorders franchise within Sanofi Genzyme, the specialty care global business unit of Sanofi.

Cablivi targets von Willebrand factor (vWF), a protein in the blood involved in hemostasis. It is designed to inhibit the interaction between vWF and platelets. Cablivi is an anti-vWF Nanobody therapeutic protein based on single-domain antibody fragments and Sanofi’s first Nanobody-based medicine to receive approval in the US. Nanobodies are a class of proprietary therapeutic proteins based on single-domain antibody fragments that contain the structural and functional properties of naturally occurring heavy-chain only antibodies.

Cablivi was approved in the European Union in August 2018. Sanofi says that Cablivi is expected to be available in the US late in the first quarter of 2019. The US list price, or wholesale acquisition cost, for treating a typical aTTP episode with Cablivi is $270,000, according to information from the company. Sanofi will be launching Cablivi Patient Solutions, a patient- support program.

In addition to Cablivi, with the acquisition of Ablynx, Sanofi gained a pipeline of more than 45 proprietary and partnered Nanobody candidates for a range of therapeutic areas such as hematology, inflammation, immuno-oncology, and respiratory diseases.

Source: Sanofi and FDA

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